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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05076617




Registration number
NCT05076617
Ethics application status
Date submitted
30/09/2021
Date registered
13/10/2021

Titles & IDs
Public title
A Study to Test the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
Scientific title
An Open-Label, Multicenter, Outpatient Extension Study to Evaluate the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
Secondary ID [1] 0 0
2021-002637-42
Secondary ID [2] 0 0
EP0165
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Stereotypical Prolonged Seizures 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Staccato alprazolam

Experimental: Staccato alprazolam - Participants will receive Staccato alprazolam by inhalation.


Treatment: Drugs: Staccato alprazolam
* Pharmaceutical form: Inhalation powder
* Route of administration: Inhalation

Participants will receive Staccato alprazolam during the Treatment Period.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of treatment-emergent adverse events (TEAEs)
Timepoint [1] 0 0
From Baseline up to the End of Study Visit (up to 48 months)
Primary outcome [2] 0 0
Frequency of TEAEs leading to withdrawal from study
Timepoint [2] 0 0
From Baseline up to the End of Study Visit (up to 48 months)
Primary outcome [3] 0 0
Frequency of serious TEAEs
Timepoint [3] 0 0
From Baseline up to the End of Study Visit (up to 48 months)
Secondary outcome [1] 0 0
Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months
Timepoint [1] 0 0
From start of IMP treatment up to 12 months
Secondary outcome [2] 0 0
Treatment success after IMP administration with no recurrence after 2 hours for seizures during the first 12 months
Timepoint [2] 0 0
From start of IMP treatment up to 12 months
Secondary outcome [3] 0 0
Frequency of respiratory TEAEs
Timepoint [3] 0 0
From Baseline up to the End of Study Visit (up to 48 months)

Eligibility
Key inclusion criteria
Inclusion criteria:

* Participant must be =12 years of age at the time of signing informed consent
* Participant must have a study caregiver =18 years of age at the time of signing the informed consent; the study caregiver(s) must be able to recognize and observe the participant's seizures
* Participants with an established diagnosis of focal or generalized epilepsy or combined focal and generalized epilepsy with a documented history of stereotypical episodes of prolonged seizures that includes at least 1 of the following:

1. Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
2. Episodes of a focal seizure with a minimum duration of 3 minutes
3. Episodes of a focal seizure or a flurry of myoclonic seizures for at least 90 seconds followed by a generalized/bilateral tonic-clonic seizure with a minimum total duration of 3 minutes
* Prior to the Screening Visit, participant completed a study using Staccato alprazolam (such as EP0162 (NCT05077904), ENGAGE-E-001 (NCT03478982), or UP0100 (NCT04857307))
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participant has a current history of alcohol or drug use disorder, as defined in the Diagnostic and Statistical Manual of Mental Disorders 5, within the previous 1 year
* Participant has a known hypersensitivity to any components of the investigational medicinal product (IMP) or comparable drugs (and/or an investigational device) as stated in this protocol or to albuterol (or similar bronchospasm rescue medication if needed to meet country-specific requirements)
* Participant has a history of convulsive (generalized tonic-clonic) status epilepticus in the 8 weeks prior to the Screening Visit
* Participant has a history or presence of known nonepileptic seizures which cannot be distinguished from qualifying epileptic seizures
* Participant has a clinically significant known airway hypersensitivity (eg, bronchospasm to known allergens, such as pollen, animals, or food) and/or acute respiratory signs/symptoms (eg, shortness of breath, wheezing on lung auscultation). NOTE: Participants with mild asthma who qualify for inclusion in the are allowed to be enrolled even though they have known airway hypersensitivity
* Participant has a clinically significant chronic pulmonary disorder other than mild asthma (eg, chronic obstructive pulmonary disease, restrictive lung diseases [including idiopathic pulmonary fibrosis]) and/or recent history or presence of hemoptysis or pneumothorax
* Participant has had a positive antigen test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and experienced moderate to severe signs/symptoms of respiratory distress necessitating hospitalization or outpatient treatment such as ambulatory oxygen, extensive treatment with inhaler medications, and/or oral medications for a duration of 4 weeks or more, unless full resolution occurred at least 6 months prior to Screening
* Participant has experienced a severe upper respiratory tract infection within 4 weeks or severe bronchitis/pneumonia within 3 months before the Screening Visit
* Participant has a history or presence of acute narrow-angle glaucoma
* Participant has a condition for which oral alprazolam is contraindicated
* Participant is taking any drug that is a strong CYP3A4 inhibitor, including azole antifungal agents (ketoconazole and itraconazole) and nefazodone
* Participant is taking any opioids or sedative hypnotics on a chronic basis
* Participant is taking nonselective beta blockers on a chronic basis

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/02/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/11/2028
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Ep0165 30016 - Fitzroy
Recruitment hospital [2] 0 0
Ep0165 30030 - Herston
Recruitment hospital [3] 0 0
Ep0165 30027 - Melbourne
Recruitment hospital [4] 0 0
Ep0165 30031 - South Brisbane
Recruitment postcode(s) [1] 0 0
- Fitzroy
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
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Arkansas
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California
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United States of America
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Connecticut
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District of Columbia
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United States of America
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Florida
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United States of America
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Hawaii
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United States of America
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Idaho
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United States of America
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Illinois
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Indiana
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Kentucky
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Louisiana
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Massachusetts
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Missouri
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New Jersey
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New York
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North Carolina
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Ohio
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Pennsylvania
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Tennessee
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Texas
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United States of America
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Utah
Country [23] 0 0
Bulgaria
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Blagoevgrad
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Bulgaria
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Pazardzhik
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Bulgaria
State/province [25] 0 0
Pleven
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Bulgaria
State/province [26] 0 0
Sofia
Country [27] 0 0
China
State/province [27] 0 0
Beijing
Country [28] 0 0
China
State/province [28] 0 0
Changchun
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China
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Chengdu
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China
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Fuzhou
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China
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Guangzhou
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China
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Hangzhou
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China
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Kunming
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China
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Lanzhou
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China
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Nanjing
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China
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Shanghai
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China
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Shijiazhuang
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China
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Suzhou
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China
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Tianjin
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China
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Wenzhou
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China
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Yinchuan
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China
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Zhanjiang
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China
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Zhengzhou
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Czechia
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Brno
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Czechia
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Ostrava - Poruba
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Czechia
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Praha 4
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Czechia
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Praha 5
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Czechia
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Praha 6
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Germany
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Berlin
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Germany
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Bielefeld
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Germany
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Erlangen
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Germany
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Frankfurt
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Germany
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Kehl
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Germany
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Marburg
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Germany
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München
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Hungary
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Balassagyarmat
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Hungary
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Budapest
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Hungary
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Debrecen
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Italy
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Genova
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Italy
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Milano
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Italy
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Pavia
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Italy
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Roma
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Japan
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Fukuoka
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Japan
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Hamamatsu
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Hiroshima
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Hofu
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Japan
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Itami
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Japan
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Kodaira
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Japan
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Koshi
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Japan
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Nagakute
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Niigata
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Omura
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Japan
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Osaka
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Japan
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Sapporo
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Japan
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Shinjuku-ku
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Japan
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Shizuoka
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Japan
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Suita
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Japan
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Toon
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Japan
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Yamagata
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Poland
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Bydgoszcz
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Poland
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Gdansk
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Poland
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Krakow
Country [83] 0 0
Poland
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Lublin
Country [84] 0 0
Poland
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Nowa Sol
Country [85] 0 0
Poland
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Swidnik
Country [86] 0 0
Spain
State/province [86] 0 0
Barcelona
Country [87] 0 0
Spain
State/province [87] 0 0
Hospitalet de Llobregat
Country [88] 0 0
Spain
State/province [88] 0 0
Madrid
Country [89] 0 0
Spain
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Pamplona
Country [90] 0 0
Spain
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Sevilla
Country [91] 0 0
Spain
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Terrassa
Country [92] 0 0
Spain
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Valencia
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Spain
State/province [93] 0 0
Valladolid
Country [94] 0 0
United Kingdom
State/province [94] 0 0
Birmingham
Country [95] 0 0
United Kingdom
State/province [95] 0 0
Cardiff
Country [96] 0 0
United Kingdom
State/province [96] 0 0
Oxford
Country [97] 0 0
United Kingdom
State/province [97] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
UCB Biopharma SRL
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
UCB Cares
Address 0 0
001 844 599 2273 (UCB)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of reidentifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
Available to whom?
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.Vivli.org


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05076617